Stargardt Disease
Conditions
Brief summary
Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis. This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.
Interventions
DRUGtinlarebant
Phase 1b Portion: tinlarebant will be self-administered orally once daily for 2 cycles, 14 days per cycle. Phase 2 portion: tinlarebant will be self-administered orally once daily for 24 months.
Sponsors
Belite Bio, Inc
RBP4 Pty Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Major Inclusion Criteria: Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene. Major
Exclusion criteria
Any ocular disease other than Stargardt disease at baseline that, in the opinion of the PI, would complicate assessment of a treatment effect.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate systemic and ocular safety and tolerability of tinlarebant. | From baseline to 24 months | To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events. |
| The optimal dose for Phase 2. | Up to 24 months | To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease. |
Secondary
| Measure | Time frame |
|---|---|
| Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma. | Up to 24 months |
| Half-life (t1/2) of tinlarebant in plasma. | Up to 24 months |
| Change in atrophic lesion size. | From baseline to 24 months. |
| Minimum concentration of RBP4 (Cmin) | Up to 24 months |
| Time to minimal plasma RBP4 level (Tmin) | Up to 24 months |
| Maximum Plasma Concentration (Cmax) of tinlarebant in plasma. | Up to 24 months |
Countries
Australia, Taiwan
Outcome results
None listed